PB2311: LOW-DOSE PREDNISON AND ELTROMBOPAG COMBINATION AS FRONTLINE TREATMENT IN NEWLY DIAGNOSED ITP PATIENTS
Authors: S.-Y. Liu, C.-Y. Yuan, X.-H. Sui, X. Liu, Y. Li, N.-N. Shan
Journal: HemaSphere (2022)

Background: Immune thrombocytopenia (ITP) is an acquired autoimmune disease characterized by isolated low platelet counts due to platelet autoimmune destruction and megakaryocytogenesis defects. The most routine first-line treatments are glucocorticoid and IVIG, while patients with severe refractory ITP are often resistant to glucocorticoid or IVIG treatment and respond to TPO-RAs. And the increase of ineffective megakaryocytes may be due to insufficient circulating TPO. Eltrombopag is approved as an orally available TPO-RAs for ITP, which efficiently induces recovery of platelet counts by stimulating the production of megakaryocytes at the level of the hematopoietic stem cell. Aims: In order to further explore the combined application of eltrombopag and first-line drugs, the efficacy and recurrence rate of the combination of eltrombopag and prednisone in the treatment of ITP were evaluated in our experiment. Methods: We performed a retrospective study to assess the response to prednison (dose 0.5-1 mg/kg/day with taper) in combination with eltrombopag (dose 25 mg/day) in 11 patients with newly diagnosed ITP (age ≥ 17 years, the median age was 45.2 years, nine patients (81.8%) were women). None of the patients had been treated with glucocorticosteroids prior to recording. Participants should be excluded if they have drug-related thrombocytopenia, are seropositive for HCV, HIV, and HBV, have an active infection, or have other malignant diseases. Early response was defined as an increased platelets response at 1 week. (R, platelets ≥ 30 × 109 / L, with at least doubling baseline; CR, platelets ≥ 100 × 109 / L) Results: Eleven consecutive patients were enrolled, and the therapeutic effect was shown in Table 1. The median basal platelet account at diagnosis was 11 × 109/L (range 2-37). All patient achieved early response (R or CR) with prednison and eltrombopag at 8 days, eight (72.7%) achieved CR, and three (40%) achieved R. CR at 1 months (platelets≥100×109/ L) was achieved in 81.8% of patients. Approximately 88.9% of patients, excluding two unrecorded persons, achieved CR beyond 3 months. Duration of prednisone was 69.1 days (range 43-150). No obvious adverse reactions occurred during the treatment. Five patients (45.5%) had achieved treatment-free remission (TFR) and median follow-up was 5 months (range 1-10), one patient relapsed to follow-up 1 month after response evaluation. Currently, nine (81.8%) patients remain in CR, one (9.1%) in R, with an additional 9.1% relapsing. Image:Summary/Conclusion: The combination of low-dose prednison and eltrombopag have the benefit of a higher early response (R or CR) rate inducing a more rapid increase in platelet count than corticosteroids alone, and usually demonstrate a more durable response. The response was also relatively lasting in follow-up after drug withdrawal. Low-dose prednisone have fewer side effects and are particularly suitable for women and older adults who cannot tolerate excessive corticosteroids. And low-dose eltrombopag also have high safety and tolerability. Thus, prednison/eltrombopag has been used as frontline therapy in ITP.